Neurofibromatosis type 1 (NF1) is a common genetic disease affecting 1 in 3500 people, regardless of race or ethnicity. NF1 is characterized by a variety of benign lesions affecting many different organ systems, as well as an increased risk for several malignancies. NF1 is defined clinically by the presence of neurofibromas (benign tumors of peripheral nerves), cafe au lait patches (pigmented skin spots), Lisch nodules (iris hamartomas), optic pathway gliomas, abnormal bone development and impaired bone healing, cardiovascular problems such as heart valve and blood vessel abnormalities, as well as cognitive and learning disabilities. NF1 patients are at an increased risk for developing multiple tumor types, including malignant peripheral nerve sheath tumors, astrocytomas/glioblastomas, pheochromocytomas, and leukemia. Many of the common benign features of NF1, such as cafe au lait patches and neurofibromas, can be extremely disfiguring leading to a greatly reduced quality of life. Neurofibromas can also cause extreme pain that is very difficult to alleviate. The malignant tumors associated with NF1 are often incurable and the life expectancy for NF1 patients is significantly reduced compared to the general population. Many of these manifestations of NF1 affect very young children and require repeated and painful surgeries over the course of their childhoods and into adulthood. Because NF1 is common, results in increased mortality, results in decreased quality of life, and no treatments are currently available, there is an unmet need for the identification of a class of NF1-specific drugs for NF1 therapy.